Orphan designation: 4-[[(1S,4S)-5-[[4-[4-(oxazol-2-yl)phenoxy]phenyl]methyl]-2,5-diazabicyclo[2.2.1]hept-2-yl]methyl]benzoic acid, Treatment of cystic fibrosis

Case Medical Research ◽

10.31525/cmr-bc10f3 ◽

2019 ◽

Author(s):

Keyword(s):

Cystic Fibrosis ◽

Benzoic Acid ◽

Acid Treatment ◽

Orphan Designation

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Orphan designation: 4-[[(1S,4S)-5-[[4-[4-(oxazol-2-yl)phenoxy]phenyl]methyl]-2,5-diazabicyclo[2.2.1]hept-2-yl]methyl]benzoic acid, Treatment of cystic fibrosis

Case Medical Research ◽

10.31525/cmr-bb17ad ◽

2019 ◽

Author(s):

Keyword(s):

Cystic Fibrosis ◽

Benzoic Acid ◽

Acid Treatment ◽

Orphan Designation

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Orphan designation: Sinapultide, dipalmitoylphosphatidylcholine palmitoyl-oleoyl phosphatidylglycerol, sodium salt and palmitic acid, Treatment of cystic fibrosis

Case Medical Research ◽

10.31525/cmr-134bca6 ◽

2019 ◽

Author(s):

Keyword(s):

Cystic Fibrosis ◽

Palmitic Acid ◽

Sodium Salt ◽

Acid Treatment ◽

Orphan Designation

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Orphan designation: Alpha-1 antitrypsin (inhalation use), Treatment of cystic fibrosis

Case Medical Research ◽

10.31525/cmr-110cf5b ◽

2019 ◽

Author(s):

Keyword(s):

Cystic Fibrosis ◽

Orphan Designation ◽

Alpha 1 Antitrypsin

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Orphan designation: Alpha-1 antitrypsin (inhalation use), Treatment of cystic fibrosis

Case Medical Research ◽

10.31525/cmr-1103412 ◽

2019 ◽

Author(s):

Keyword(s):

Cystic Fibrosis ◽

Orphan Designation ◽

Alpha 1 Antitrypsin

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Orphan designation: Multilamellar microvesicle comprising phosphatidylcholine, sphingomyelin, phosphatidylethanolamine, phosphatidylserine, phosphatidylinositol and cholesterol, Treatment of cystic fibrosis

Case Medical Research ◽

10.31525/cmr-22295fd ◽

2019 ◽

Author(s):

Keyword(s):

Cystic Fibrosis ◽

Orphan Designation ◽

Cholesterol Treatment

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Orphan designation: Recombinant human acid ceramidase, Treatment of cystic fibrosis

Case Medical Research ◽

10.31525/cmr-1f6761d ◽

2019 ◽

Author(s):

Keyword(s):

Cystic Fibrosis ◽

Acid Ceramidase ◽

Orphan Designation ◽

Human Acid

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Orphan designation: (6aR,10aR)-3-(1,1-dimethylheptyl)-delta8-tetrahydro-cannabinol-9-carboxylic acid, Treatment of dermatomyositis

Case Medical Research ◽

10.31525/cmr-863651 ◽

2019 ◽

Author(s):

Keyword(s):

Carboxylic Acid ◽

Acid Treatment ◽

Orphan Designation

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Orphan designation: 6,8-bis(benzylthio)octanoic acid, Treatment of acute myeloid leukaemia

Case Medical Research ◽

10.31525/cmr-d6cc8d ◽

2019 ◽

Author(s):

Keyword(s):

Acute Myeloid Leukaemia ◽

Myeloid Leukaemia ◽

Acid Treatment ◽

Octanoic Acid ◽

Orphan Designation ◽

Acute Myeloid

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Orphan designation: 6,8-bis(benzylthio)octanoic acid, Treatment of acute myeloid leukaemia

Case Medical Research ◽

10.31525/cmr-d6788f ◽

2019 ◽

Author(s):

Keyword(s):

Acute Myeloid Leukaemia ◽

Myeloid Leukaemia ◽

Acid Treatment ◽

Octanoic Acid ◽

Orphan Designation ◽

Acute Myeloid

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Orphan designation: N-(2,4-di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide (ivacaftor), Treatment of cystic fibrosis

Case Medical Research ◽

10.31525/cmr-58722a ◽

2018 ◽

Author(s):

Keyword(s):

Cystic Fibrosis ◽

Orphan Designation ◽

Tert Butyl

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